De Sanctis V, Galimberti M, Lucarelli G, Polchi P, Ruggiero L, Vullo C
Department of Paediatrics, Arcispedale S Anna, Ferrara.
Arch Dis Child. 1991 Apr;66(4):517-20. doi: 10.1136/adc.66.4.517.
Thirty prepubertal patients with thalassaemia major (15 boys and 15 girls) aged from 9.3 to 17.2 years (mean 12.9) who had successfully undergone allogenic bone marrow transplantation were studied. Before the transplant all patients were given short courses of high doses of busulphan (total dose 14 mg/kg) followed by cyclophosphamide (total dose 200 mg/kg). Pituitary gonadal function was assessed between 0.7 and 5.1 years (mean 2.3) after bone marrow transplantation. Increased gonadotrophin concentrations indicating gonadal damage were found in 80% of the girls, probably as a result of the chemotherapy. In all the prepubertal boys the basal follicle stimulating hormone and luteinising hormone concentrations were normal. Most of the boys had reduced gonadotrophin and testosterone responses after gonadotrophin releasing hormone and human chorionic gonadotrophin tests. This could have been the result of iron overload but the effect of cytotoxic agents cannot be excluded. These findings emphasise the need for vigilant long term follow up of thalassaemic patients treated with cytotoxic chemotherapy for bone marrow transplantation so that those requiring hormone replacement can be identified and treated.
对30例年龄在9.3至17.2岁(平均12.9岁)之间成功接受异基因骨髓移植的重型地中海贫血青春期前患者(15名男孩和15名女孩)进行了研究。移植前,所有患者均接受了短疗程高剂量白消安(总剂量14mg/kg),随后给予环磷酰胺(总剂量200mg/kg)。在骨髓移植后0.7至5.1年(平均2.3年)评估垂体性腺功能。80%的女孩出现促性腺激素浓度升高,提示性腺受损,这可能是化疗的结果。所有青春期前男孩的基础促卵泡生成素和促黄体生成素浓度均正常。大多数男孩在促性腺激素释放激素和人绒毛膜促性腺激素试验后促性腺激素和睾酮反应降低。这可能是铁过载的结果,但细胞毒性药物的影响也不能排除。这些发现强调了对接受细胞毒性化疗进行骨髓移植的地中海贫血患者进行长期密切随访的必要性,以便识别和治疗那些需要激素替代治疗的患者。